The overall survival for children with high risk NBL is poor and roughly 20-35%.
The disease is generally very responsive to chemotherapy, but there is a high relapse rate.
The standard treatment is generally regarded as:
Phase 1= Induction with:
- Resection of primary tumor attempted after chemotherapy response
- Stem cell harvest
Phase 2 = Consolidation with:
- Hematopoietic stem cell rescue or transplantation (HSCT)
- Conditioning regimen is generally carboplatin/etoposide/melphalan or busulfan/melphalan
- TBI (low dose whole body radiation therapy) no longer used with the aim of reducing long term side effects
- Radiation therapy to primary site after transplant to reduce risk of local recurrence
Rapid, sequential, tandem myeloablative consolidation therapy may improve the outcome
Phase 3 = Post consolidation with:
- Antibodies to target GD2 present on the surface of NBL cells used i.e. chimeric anti-GD2 antibody ch14.18 combined with GM-CSF and interleukin-2 given with the retinoic acid improve event free survival
- Oral 13-cis-retinoic acid for 6 months
COG-ANBL0532: This study is now closed to accrual. The aim was to improve event free survival (EFS). This protocol used:
- Dose intensification of the induction chemotherapy (with addition of dose-intensive topotecan and cyclophosphamide in induction)
- Randomized substitution of two cycles of topotecan and cyclophosphamide for two cycles of vincristine, cyclophosphamide, and doxorubicin to assess if this improved outcome.
- Tandem transplant using marrow support
- Randomization between two cycles of myeloablative chemotherapy and stem cell transplantation vs a single cycle of myeloablative chemotherapy and stem cell transplantation.
- Escalation of RT dose to improve local control
COG-ANBL0032 and COG-ANBL0931: COG non-randomized study to look at the efficacy of monoclonal antibody therapy with granulocyte-macrophage colony-stimulating factor and interleukin-2 combined with cis-retinoic acid following completion of chemotherapy
The New Approaches to Neuroblastoma Therapy (NANT) consortium is studying inclusion of myeloablative doses of iodine-131-meta-iodobenzylguanidine (MIBG) with myeloablative chemotherapy prior to stem cell transplantation in patients with an incomplete response to induction chemotherapy.